Akcea receives their first drug approval from the European Commission

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Ottawa-based Akcea Therapeutics Inc. receives their first drug approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR. 

“With the EC’s decision, Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis,” says Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “With subcutaneous delivery, Tegsedi puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe Tegsedi enables people and their families impacted by this disease to move forward with their lives.

Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch Tegsedi along with our patient and physician support services across Europe,” she adds.

The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Tegsedi is designed to block the production of the TTR protein. In the NEURO-TTR study, treatment with Tegsedi produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of the disease.

“hATTR amyloidosis is an inherited, progressive and fatal disease for which treatment options are limited. The approval of TEGSEDI brings us into a new era of treatment with an efficacious and disease-modifying medicine that potentially allows patients to achieve a greater degree of independence,” says Teresa Coelho, M.D., neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. “TEGSEDI has demonstrated rapid and sustained benefits in improving the course of this disease and preserving quality of life.”

The European Commission’s approval of Tegsedi was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The study demonstrated that patients treated with Tegsedi experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.

“Today, we are thrilled to see our successful research and development efforts result in the approval of an important new medicine for patients with hATTR amyloidosis. Using our antisense technology platform, we set out to design a therapy to block the production of the underlying cause of this disease, the TTR protein,” says Brett P. Monia, Ph.D., chief operating officer at Ionis Pharmaceuticals, an affiliate of Akcea Therapeutics.

“Approval of Tegsedi further establishes Ionis as a multi-product company. We are confident that the experienced team at Akcea will deliver on the promise of TEGSEDI. We are grateful to all of the physicians and patients who participated in the TEGSEDI clinical program and who made this landmark approval possible,” he adds.

Health Canada granted priority review for the New Drug Submission (NDS) filed for Tegsedi and is anticipating approval at some point this year.

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